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Trial intelligence / Profile preview
Avalglucosidase alfa in non-classic Pompe disease (≥5 years)
2024-518215-18-00Phase 4Authorised
- Sponsor
- Erasmus MC
- Start
- Oct 2024
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 1
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Overview
Phase 4, open-label, single-arm trial of avalglucosidase alfa in Pompe disease, sponsored by Erasmus MC. Primary endpoints: Safety and tolerability, Pharmacokinetics of avalglucosidase alfa and 4 more. Planned start Oct 2024; status: authorised.
02
Drugs
avalglucosidase alfa
03
Conditions
Pompe disease
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Competitive context
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